FDA awards two grants for natural history studies in rare diseases

October 8, 2019

For Immediate Release:

October 08, 2019

The U.S. Food and Drug Administration today announced it has awarded two new research grants for natural history studies in rare diseases. The FDA is providing over $4.1 million over the next four years to fund these studies. Information from natural history studies can facilitate design of efficient clinical trials to test future treatments.

Natural history studies closely look at how specific diseases progress over time. The natural history of a disease is the course a disease takes from its onset, through the presymptomatic and clinical stages, to a final outcome in the absence of treatment.

“The FDA is committed to funding these important studies in order to define how rare diseases develop and progress,” said FDA Principal Deputy Commissioner Amy Abernethy, M.D., Ph.D. “Further, these studies provide important roadmaps for how to conduct subsequent studies. With the natural history of many rare diseases remaining relatively unknown, efficiently developing diagnostics and therapies for patients poses unique challenges. Promoting and conducting work in this area is critical.”

The FDA received 31 grant applications that were reviewed and evaluated for scientific and technical merit by more than 45 rare disease, natural history, regulatory and statistical experts, that included representatives from academia, patient groups, the National Institutes of Health and the FDA. The grants were awarded to:

University of Texas MD Anderson Cancer Center (Houston, Texas), Elizabeth Grubbs, prospective study in medullary thyroid carcinoma, approximately $1.7 million over four years

The study seeks to leverage a multi-institutional registry to characterize disease variables and patient perspectives that inform decisions regarding initiation of and adherence to chemotherapy in medullary thyroid cancer. Such critical knowledge can be incorporated into the design of clinical trials of emerging therapies for this disease.

Vanderbilt University Medical Center (Nashville, Tennessee), Jonathan Soslow, prospective study in cardiac disease in Duchenne muscular dystrophy, approximately $2.4 million over four years.

This study aims to focus on cardiomyopathy (heart muscle disease), which is the leading cause of death in Duchenne muscular dystrophy. The study will combine genetic differences with imaging and blood biomarkers to identify surrogate biomarkers that predict the risk of cardiac dysfunction in Duchenne muscular dystrophy and other related diseases. This information has the potential to improve future clinical trial efficiency in these diseases by decreasing their size and cost.

Congress appropriates funding to the FDA for the study of rare diseases. The FDA uses these funds for the Orphan Products Grants Program to support these natural history studies as well as clinical trials for rare diseases.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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